Study to Evaluate Safety and Efficacy of Different PANZYGA Dose Regimens in Pediatric Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) Patients

• Status: Not yet recruiting
• Trial end date: 2025-06-01
• Target enrollment: 0
• Participant gender: All

Summary

Safety and Efficacy of Different PANZYGA Dose Regimens in Pediatric Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) Patients

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Octapharma

Criteria

Inclusion Criteria:

1. Age ≥2 years and ≤17 years.

2. Patients with a diagnosis of definite or probable CIDP based on European Neuromuscular Center (ENMC) criteria.

3. Clinical history of functional impairment due to CIDP, corresponding to an mRS score ≥2, but ≤5.

4. Voluntarily given written informed consent (provided by patient's parent or legal guardian) or assent (provided by patient, if age-appropriate per Independent Ethics Committee [IEC]/Institutional Research Board [IRB] requirements).

Exclusion Criteria:

1. Patients with previously diagnosed CIDP who lack any CIDP symptoms.

2. Patients with a known history of inherited neuropathy or a family history of inherited neuropathy.

3. Patients who have previously failed immunoglobulin therapy for CIDP.

4. Patients who received immunoglobulin within 8 weeks prior to the Baseline visit (washout phase). However, if a patient has clinical evidence of confirmed CIDP relapse during the washout phase (consistent with an increase in mRS of ≥1), they are eligible for trial enrolment.

5. Patients with a history of deep vein thrombosis (DVT) in the past year, or pulmonary embolism ever.

6. Patients on unstable (change in prescribed dose within the last 8 weeks) corticosteroids or rituximab use.

7. Patients with known or suspected hypersensitivity, anaphylaxis or severe systemic response to immune-globulins, blood or plasma derived products, or any component of PANZYGA.

8. Female patients who are breastfeeding, pregnant, or planning to become pregnant, or are unwilling to use an effective birth control method while on the study (acceptable methods of birth control for this study include: intrauterine device [IUD], hormonal contraception, male or female condom, spermicide gel, diaphragm, sponge, or cervical cap).

9. Known human immunodeficiency virus (HIV), hepatitis B virus (HBV), and/or hepatitis C virus (HCV) infections.

10. Severe liver and/or kidney disease (alanine aminotransferase [ALT] > 3 × upper limit of normal [ULN]; aspartate aminotransferase [AST] > 3 × ULN; and/or creatinine levels >44 μmol/L for children ages 2-3 years, >62 μmol/L for children ages 4-10, and >89 μmol/L for children ages 11-17 years.

11. Known immunoglobulin (IgA) deficiency and antibodies against IgA.

12. History of alcohol or drug abuse in the previous year, as per Investigator's opin-ion.

13. Unable or unwilling to comply with the study protocol.

14. Receipt of any other investigational medicinal product (IMP) within 3 months be-fore study entry.

15. Any other condition(s) that, in the Investigator's opinion, makes it undesirable for the patient to participate in the study or may interfere with protocol compliance.